Donate

Industry News

Genentech Presents Latest Data on Emicizumab (ACE910)

July 29, 2016

Note: The below is an edited version of an article originally published on BusinessWire.聽 The original article can be read in their entirety聽here. The latest data from an ongoing Japanese Phase I/II study of Emicizumab were presented earlier this week at the World Federation of Hemophilia 2016 World Congress.聽 Emicizumab (a product being developed by […]

BioMarin Releases Results of Phase 1/2 Study of Gene Therapy Treatment

July 28, 2016

Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety聽here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]

Sangamo BioSciences Announces New Gene Therapy Clinical Development Program

July 28, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its聽entirety聽here. Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated […]

uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B

July 28, 2016

Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety聽here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.聽 The updated data show that […]

CSL Behring Presents Phase 3 Data about Afstyla for Hemophilia A

July 27, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in it’s entirety聽here. CSL Behring聽today presented data from a Phase III pivotal study of Afstyla聽[Antihemophilic Factor (Recombinant), Single Chain] in hemophilia A patients less than 12 years of age at the XXXII International Congress […]

Voluntary Withdrawals of Helixate FS & Kogenate FS

July 26, 2016

As part of HFA鈥檚 ongoing mission to assist and advocate for the bleeding disorders community, this medical news is being issued to keep you informed about product safety. About These Withdrawals CSL Behring is initiating a voluntarily recall of two lots of its hemophilia A drug Helixate FS containing active ingredient manufactured before November 2015. […]

Adynovate Phase 3 Efficacy and Safety Data in Children to be Presented

July 26, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in it’s entirety here. _________________________________________________________________________________________ Shire will present additional data on the clinical experience of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended half-life recombinant Factor VIII (rFVIII) replacement for hemophilia A, during the International […]

CSL Behring Presents Phase III Data for IDELVION for Hemophilia B

July 26, 2016

Note: The below is an edited version of an article originally published on Yahoo Finance. The original release can be read in it’s entirety here. _________________________________________________________________________________________ CSL Behring announced new data today from its Phase III PROLONG-9FP clinical development program evaluating聽IDELVION庐 [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], the company’s long-acting recombinant albumin fusion protein […]

Alnylam Announces New Positive Interim Phase 1 Study Results for Fitusiran

July 26, 2016

Note: The below is an edited version of an article originally published on StreetInsider.com. The original release can be read in it’s entirety聽here. _________________________________________________________________________________________ Alnylam Pharmaceuticals, Inc. announced today new positive results from its ongoing Phase 1 study with Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B and […]

Novo Nordisk Announces New Data on Psychosocial Impact of People Living with Hemophilia B

July 26, 2016

Note: The below is an edited version of a press release by Novo Nordisk. The original release can be read in it’s entirety聽here.聽 Novo Nordisk today announced the first results from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study, which found that many adults and children living with even mild or […]

Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B

July 21, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. Spark Therapeutics and Pfizer Inc. announced today that the聽U.S. Food and Drug Administration聽(FDA) has granted breakthrough therapy designation to聽SPK-9001,聽the lead investigational candidate in the companies’聽SPK-FIX聽program, in development for the treatment of hemophilia B.聽SPK-9001,聽a […]

Kedrion Announces Voluntary, Temporary Hold on Koate-DVI

July 6, 2016

Kedrion Biopharma provided the following announcement today: Koate庐-DVI Summer Supply Statement Kedrion Biopharma has placed a voluntary, temporary hold on further shipments聽of Koate-DVI until approximately the September timeframe. The company is working to restore supplies of Koate-DVI as soon as possible so聽that it can be made available to patients who require it. This is being […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]

Spark Therapeutics Announces Updated Data from Hemophilia B Trial

June 14, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. PHILADELPHIA,聽June 13, 2016 –聽Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of聽SPK-9001, the lead investigational candidate in its聽SPK-FIX聽program, which is being studied for the […]

Shire Completes Combination with Baxalta

June 3, 2016

Today, June 3, 2016, Shire completed its previously announced acquisition with Baxalta Incorporated. The combined company is expected to deliver over $20 billion in annual revenues by 2020. The聽company is focused on serving patients with rare diseases and other highly specialized conditions. Read the press release from Shire in it’s entirety here.   About Shire聽 […]

FDA Approves First Single Chain Product for Hemophilia A

May 27, 2016

Note: The below is an edited version of a press release by CSL Behring.聽The original release can be read in it’s entirety聽here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]

Biogen Announces Intent to Spin off Its Hemophilia Business

May 3, 2016

Note: The below is an edited version of a press release from Biogen Inc. The original release can be read in its entirety here. Biogen Inc. announced on Tuesday, May 3, 2016 that it intends to spin off its hemophilia business as an independent, publicly traded company. The strategic goal of this transaction is to […]

FDA Approves Needle-Free Transfer Device for Hemophilia A Product

March 31, 2016

Note: The following in an edited press release from Kedrion Biopharma. The original release can be read in its entirety here. Kedrion Biopharma has gained approval from the U.S. Food and Drug Administration to package聽Ko膩te庐 Double Viral Inactivation (DVI) Antihemophilic Factor (human)聽with Mix2Vial鈩, a needle-free transfer device. The new packaging is designed to offer hemophilia […]

FDA Approves New Recombinant Factor Following LEOPOLD Trials

March 17, 2016

Note: The following is an edited version of a press release from Bayer. The original press release can be found here.聽 The U.S. Food and Drug Administration (FDA) has approved Bayer鈥檚 KOVALTRY庐 Antihemophilic Factor (Recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based […]

FDA Approves First 14-Day Hemophilia B Treatment

March 5, 2016

Note: The following is an edited form of a press release from the FDA . The original form of the release can be read聽here. The U.S. Food and Drug Administration approved on Friday, March 4, Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the […]

FDA Gives Orphan Drug Designation for Hemophilia A Gene Therapy

March 1, 2016

Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]

FDA Approves Treatment of Chronic Hepatitis C Genotypes 1 and 4

February 1, 2016

Note: The following is an edited form of a press release from the Food and Drug Administration. The original form of the release can be read here. On Thursday, January 28, the U.S. Food and Drug Administration approved Zepatier (elbasvir and grazoprevir) with or without ribavirin for the treatment of chronic hepatitis C virus (HCV) […]

Future Bisomilars: Pros and Cons

January 20, 2016

Note: This story was originally published in the January 2016 issue of BioSupply Trends Quarterly. It can be read in its original format here. By Meredith Whitmore In this brave new world of medicines, more and more physicians are prescribing a biologic. Perhaps they have even witnessed a patient鈥檚 remarkable transformation thanks to biologics such […]

Drugmaker Shire Wins Baxalta for $32 Billion After Six-Month Pursuit

January 11, 2016

Note: The following story was published by Reuters聽on Monday, January 11, 2016 . It can be found here. Drugmaker Shire Plc clinched its six-month pursuit of Baxalta International Inc on Monday with an agreed $32 billion cash and stock offer, catapulting it to a leading position in treating rare diseases. The London-listed group, which first […]

uniQure Announces Preliminary Results from Hemophilia B Gene Therapy Trial

January 7, 2016

Note: the following is the 聽edited form of a 聽press releaseoriginally published by uniQure. The full release can be read here.聽 On Thursday, January 7, 2016, uniQure N.V. announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure鈥檚 novel AAV5/FIX […]

Octapharma Announces US Availability of NUWIQ

January 4, 2016

聽Note: The following is an edited press release from Octapharma USA. The original release can be read in its entirety聽here. Octapharma USA announced on Monday, January 4, 2016 that NUWIQ庐, Antihemophilic Factor (Recombinant), is now commercially available. NUWIQ庐 is indicated for the treatment and control of bleeding, perioperative (surgical) management, and routine prophylaxis to reduce […]

European Commission Grants $6M to Develop New Hemophilia A Treatment

December 29, 2015

Note: The following is an edited press release from Sernova Corp. The original release can be read in its entirety here. Sernova Corp., a clinical stage regenerative medicine company, announced on Monday, December 21, 2015, that the European Commission鈥檚 Horizon 2020 program has awarded a Euro 5.6M ($6.1M USD) grant to a consortium consisting of […]

Arsia and Biogen Team Up to Develop Sub-Q Hemophilia Treatment

December 16, 2015

Note: The following is an edited press release from Arsia Therapeutics, Inc. The original release can be read here. Arsia Therapeutics, Inc., a Cambridge based company, announced a collaboration with Biogen on December 16, 2015. Arsia and Biogen will focus on providing meaningful treatment administration improvements for hemophilia patients by enabling subcutaneous versions of treatments […]

FDA Approves First Recombinant von Willebrand Factor

December 8, 2015

The U.S. Food and Drug Administration today approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand (as needed) treatment and control of bleeding episodes in adults diagnosed […]

Phase I Study Results for Once-Monthly Subcutaneous Hemophilia Treatment

December 8, 2015

Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]


Sign up for E-mails, Dateline Magazine, and other ways to stay connected.